首页> 外国专利> EXOSOME LOADED THERAPEUTICS FOR THE TREATMENT OF NON-ALCOHOLIC STEATOHEPATITIS, DIABETES MELLITUS TYPE 1 AND TYPE 2, ATHEROSCLEROTIC CARDIOVASCULAR DISEASE, AND ALPHA 1 ANTITRYPSIN DEFICIENCY

EXOSOME LOADED THERAPEUTICS FOR THE TREATMENT OF NON-ALCOHOLIC STEATOHEPATITIS, DIABETES MELLITUS TYPE 1 AND TYPE 2, ATHEROSCLEROTIC CARDIOVASCULAR DISEASE, AND ALPHA 1 ANTITRYPSIN DEFICIENCY

机译:外来负载疗法治疗非酒精性脂肪性肝炎,1型和2型糖尿病,动脉粥样硬化性心血管疾病和α1抗胰蛋白酶缺乏症

摘要

A composition for delivering a cargo to the cytoplasm of a cell, wherein the cargo treats Non-Alcoholic Steatohepatitis, Non-Alcoholic Fatty Liver Disease, Diabetes Mellitus Type 1 and Type 2, Atherosclerotic Cardiovascular Disease, and Alpha 1 Antitrypsin Deficiency. In another embodiment, the composition comprises: an exosome; cargo located inside the exosome, wherein the cargo comprises short interference RNA (siRNA) that depletes sodium-glucose linked transporter active sites. In another embodiment, the composition comprises: an exosome; cargo location inside the exosome, wherein the cargo corrects the missense SERPINA1 mutation from 'Z' to 'M'.
机译:一种用于将货物递送至细胞质的组合物,其中所述货物治疗非酒精性脂肪性肝炎,非酒精性脂肪肝病,1型和2型糖尿病,动脉粥样硬化性心血管疾病和α1抗胰蛋白酶缺乏症。在另一个实施方案中,所述组合物包含:外泌体;和位于外泌体内部的货物,其中所述货物包含耗尽钠-葡萄糖连接的转运蛋白活性位点的短干扰RNA(siRNA)。在另一个实施方案中,所述组合物包含:外泌体;和货物在外泌体内部的位置,其中货物纠正了从'Z'到'M'的错义SERPINA1突变。

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